Date: 2011-03-28
Type of information: Development agreement
Compound: gene therapy treatments for six rare genetic diseases
Company: MolMed (Italy) Fondazione Telethon (Italy)
Therapeutic area: Rare diseases - Genetic diseases
Type agreement: development
manufacturing
production
Action mechanism: gene therapy
Disease: metachromatic leukodystrophy (MLD) - Wiskott-Aldrich syndrome (WAS) - beta-thalassemia mucopolysaccharidosis type I (MPS I) - globoid leukodystrophy (GLD) - chronic granulomatous disorder (CGD)
Details: MolMed and Fondazione Telethon have signe an agreement to develop and manufacture novel gene therapy treatments for six rare genetic diseases. The six diseases involved - metachromatic leukodystrophy (MLD), Wiskott-Aldrich syndrome (WAS), beta-thalassemia, mucopolysaccharidosis type I (MPS I), globoid leukodystrophy (GLD) and chronic granulomatous disorder (CGD) - are caused by a single defective gene, making it possible to develop a potential cure by inserting the correct form of the gene into the patient’s own stem cells, derived from bone marrow, through ex vivo gene transfer technology.
Under the agreement, MolMed will develop and produce clinical grade lentiviral vectors carrying the relevant therapeutic gene and manufacture patients’ cells to be investigated in clinical trials. Studies are ongoing for MLD and WAS since 2010.
Financial terms: Under the terms of the agreement, MolMed will be receiving up to € 8.3 million in revenues over a four-year period.
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